Avrobio Plans Registration Trial With Kidney Biopsy Endpoint To Support Potential Full Approval Of Fabry Disease Gene Therapy

Avrobio Inc AVRO has provided an update on its regulatory plans for AVR-RD-01, its lentiviral gene therapy for Fabry disease, currently in FAB-GT Phase 2 trial.
In March, the FDA granted full approval to Sanofi's SA SNY Fabrazyme (agalsidase beta) after it received accelerated approval based on a surrogate endpoint of reduction of GL-3 inclusions in biopsied renal peritubular capillaries (PTCs).
The conversion of Fabrazyme to full approval limits the accelerated approval pathways available for new therapies to treat Fabry disease.
Hence, Avrobio can no longer pursue an accelerated approval pathway for AVR-RD-01 and instead plans to discuss with FDA a registration trial with a primary efficacy endpoint of clearance of GL-3/Gb3 inclusions in biopsied renal PTCs.
Registration trial is expected to initiate in mid-2022.
To support the use of AVR-RD-01 in a broad Fabry disease population, the company expects to include female patients, eliminate antibody status exclusions and collect additional cardiovascular and CNS data in its ongoing FAB-GT trial.
Price Action: AVRO shares are down 5.75% at $11.01 in the premarket trading on the last check Monday.